Sarepta Therapeutics Inc (NASDAQ:SRPT) shares traded 10% higher last week after the company released “very positive” results on its newest Duchenne muscular dystrophy (DMD) therapy called golodirsen Exon-53. DMD is a genetic disease, which causes the deterioration of muscles in young males, due to a mutation on the X chromosome, leaving many dead by the age of 20.
By using exon skipping, which tricks the cellular machinery to skip over an exon by using molecular plastering, Sarepta’s drug may provide hope as a potential treatment for a significant unmet medical need in DMD. The DMD gene is thus prevented from being pieced together and activated during the protein production stage. This is accomplished by raising dystrophin immunofluorescence and more importantly dystrophin protein by just over 1%. Notably, golodirsen’s new phase 1/2 data showed the successful deletion of the exon 53 in all 25 boys treated.
Top analyst Ritu Baral of Cowen notes, “we are very encouraged by these levels of dystrophin expression by golodirsen, we view the 1% of normal threshold to provide very strong POC for biochemical activity. These expression levels are a marked increase to the 0.28% of normal baseline corrected expression (0.44% of normal absolute) by western blot levels demonstrated by Exondys 51 […] SRPT indicated the quantification methodology used in the golodirsen Ph1/2 most closely resembled that used in the Exondys ‘4th biopsy’ analyst with a few further refinements. Nevertheless, as we had previewed in our last note, the consistency between the western blot, PCR and IHC dataset are critical to our very positive view of today’s data.”
As such, the analyst reiterates an Outperform rating on SRPT with a price target of $69.00 representing a 54% rise over current trading levels.
Additionally, looking forward, Baral highlights: “Meaningful analysis would require case matched controls as comparators. The first 6MWT analysis will be run at 96 weeks and the trial is continuing for a total of 144 weeks. SRPT did not release any safety or tolerability findings of the Ph1/2 (we will be watching LFTs and platelet levels closely despite Exondys’ excellent safety profile). The company still expects to present full data at an upcoming medical meeting, which we continue to believe could be the WMS meeting in early November.”
Ritu Baral has a solid TipRanks score with a 66% success rate and a high ranking of #66 out of 4,633 analysts. Baral realizes 20.7% in her annual returns. When recommending SRPT, the analyst posts gains of 2.2%.
Tipranks analytics reveal SRPT as a Strong Buy. Out of 13 analysts polled by TipRanks (in the past 3 months), 11 are bullish, while 2 are sidelined on Sarepta stock. The stock’s consensus price target stands at $64.78 representing a 46% rise over current trading levels.